Current Alzheimer's Disease (AD) enrolling studies with a brief description:

Study and Contact Information	Description
EnVivo (sponsored by Forum Pharmaceuticals) Protocol #: EVP-6124-025A Principal Investigator: Oleg V. Tcheremissine, MD Coordinators: Dineen Gardner – 704-446-7511 & Megan Kramer – 704-446-0803	This study is being done for the investigational drug, EVP-6124 or encenicline. “Investigational” means the drug is not approved for sale by the U.S. Food and Drug Administration or any other regulatory or health agency. The EnVivo study aims to determine if EVP-6124 is a safe and effective treatment for Alzheimer’s disease. EVP-6124 targets certain areas in the brain that contain receptors that are important for cognitive and memory functions. The study is being conducted at 60-80 centers worldwide, including CMC-Neurology. Approximately 790 people will participate for up to 34 weeks (238 days). There will be at least 10 visits to the study site as well as contacts by phone. Subjects who complete this study may have the option to participate in a separate study during which they would receive EVP-6124 for an additional 26 weeks (182 days). Individuals who do not participate in the extension study will be contacted by phone 30 days after the last time they took the study drug.

Current Amyotrophic Lateral Sclerosis (ALS) enrolling studies with a brief description:

Study and Contact Information	Description
Ibudilast in ALS (sponsored by MediciNova Inc.) Protocol #: MN-166-ALS-1201 Principal Investigator: Benjamin Brooks, MD Coordinator: Cynthia Lary – 704-446-6063	The primary goal of this study is to evaluate the safety, tolerability and effectiveness of the investigational drug ibudilast when administered as an adjunct to riluzole in people with ALS. Sixty subjects from 18 to 80 years old will be enrolled. Participants will be randomized (like flipping a coin) into 1 of 2 groups: group 1 will receive ibudilast with riluzole; group 2 will receive placebo (which looks like the ibudilast tablet, but has no active ingredients) with riluzole. This part of the study is called “double blind,” because neither the study staff nor the study patients will know which group the participants are in. After completing the double-blind phase, subjects randomized to the placebo arm will begin to receive ibudilast for an additional 6 months so that researchers can further evaluate the drug’s safety and tolerability. If researchers find no concerns, they will decide whether to extend participation to the ibudilast-treated group for an additional six months.
DPS (sponsored by MDA and ALSA) Protocol #: HUD #10-0242 Principal Investigator: Benjamin Brooks, MD Coordinator: Cynthia Lary – 704-446-6063	This study is being done to find out if the NeuRx® Diaphragm Pacing System™ (DPS) will improve breathing function or prolong life span in people with ALS. The study also aims to compare measures of diaphragm function, dyspnea and quality of life between the standard of care group and DPS group. Study participants must be at least 21. They will be randomized into one of two groups. One group (two-thirds of participants) will receive the DPS and the other group (one-third of participants) will receive standard medical care for ALS. Participants receiving standard medical care will be the “control” group in order to tell whether the effects seen are truly from the DPS.
Exercise (sponsored by Johns Hopkins University) Protocol #: NA_00022650 Principal Investigator: Benjamin Brooks, MD Coordinator: Cynthia Lary – 704-446-6063	The purpose of this study is to see whether one type of exercise is tolerated better and is safer than another for people with ALS. Researchers also will collect information about how the body responds to exercise in people with ALS. In this study, participants exercise in one of three ways: 1) weightlifting (resistance exercise); 2) stationary bicycling (endurance exercise), and 3) range of motion/stretching exercise (the “standard of care” for ALS patients). If resistance, endurance or range of motion exercise is shown to be well-tolerated and safe over 24 weeks, a larger trial will be planned to determine if exercise is beneficial to ALS patients.
MDA Patient Registry (sponsored by MDA) Protocol #: None Principal Investigator: Benjamin Brooks, MD Coordinator: Janice Rucker – 704-446-1902	This is a study for children and adults who have been diagnosed with amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) or spinal muscular atrophy (SMA). The study is an ongoing data collection effort known as the Patient Registry and sponsored by the Muscular Dystrophy Association (MDA). The Patient Registry collects data – via several questionnaires based on previous clinical assessments and patients’ own responses – from individuals with neuromuscular disease. Researchers want to better understand the disease progression and ultimately improve the care and survival of those with neuromuscular disease. Subjects’ participation is expected to last for the duration of the study, which is continuous.

Current Multiple Sclerosis (MS) enrolling studies with a brief description:

Study and Contact Information	Description
ARPEGGIO (sponsored by TEVA Pharmaceuticals) Protocol #: TV5600-CNS-20006 Principal Investigator: Jill Conway, MD Coordinator: Megan Kramer – 704-446-0803	The purpose of this study is to find out whether treatment with laquinimod is safe and effective in reducing the loss of brain volume and reducing MS worsening. Patients with PPMS may experience a reduction in their brain volume over time, and reduction in the size of the brain is thought to be related to worsening of MS. Laquinimod is a new oral drug that is being developed for the treatment of MS and other indications. It is “investigational” because it has not been approved by regulatory authorities, such as the Food and Drug. This is the first study that evaluates laquinimod in patients with PPMS. Potentially eligible participants must be between ages 25 and 55, inclusive; have a confirmed diagnosis of PPMS; show disease progression in the previous 2 years and have an EDSS limited to patients with an EDSS score of 3.0 to 5.5, inclusive. Approximately 375 patients will take part in the study at 120 sites in the U.S., Canada and Europe, including 3-5 patients at CMC-Neurology. Participation is due to last 18-30 months.
INSPIRE Protocol #: Principal Investigator: Donna Graves, MD Coordinator: Joyce Pitner – 704-446-1349	The goal of this study is to assess the efficacy of the drug BG00012 in delaying disability progression in subjects with secondary progressive multiple sclerosis (SPMS) who are progressing independent of relapses. The drug is currently marketed for the treatment of relapsing MS. This is the first trial to measure the effectiveness of BG00012 in people with SPMS. Subjects will be randomized to receive either BG00012 or placebo (which looks like the BG00012 capsule but does not have any active ingredient). Each person will have a 50-50 chance of being placed into either group. Both groups will taking one 240-mg capsule twice daily. Individuals with SPMS may be eligible for the study if they are between 18 to 58 years old, inclusive; experienced the onset of SPMS at least 2 years before; have documented, confirmed evidence of disease progression independent of clinical relapses over the previous year; have an EDSS score of 3.0 to 6.5, inclusive; have an MS Severity Score (MSSS) of 4 or higher. Study participation will last for 108 weeks.

Pending Trials

Pending studies with description

Study and Contact Information	Description
EVIDERA (sponsored by Biogen Idec) Protocol #: A13908 Principal Investigator: Benjamin Brooks, MD Coordinator: Rita Rouse – 704-446-1902	The objectives of this study are to conduct interviews to: 1) evaluate the signs, symptoms, and functional limitations that adult patients and caregivers consider important in evaluating ALS; and 2) obtain feedback on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). Patient and caregiver will take part in separate interviews during one study visit. Each interview will last 60 to 90 minutes. At CMC-Neurology, approximately 15 sets of ALS patients and their caregivers will take part in the study. The goal is to get 5 patient-caregiver pairs in each of the following ALSFRS-R score categories: 0 to 16, 17 to 32, and 33 to 48. To participate in the study, ALS patients must, among other criteria, be at least 18 and have been diagnosed with ALS 5 years ago or less. Caregivers can be a family member, medical assistant or home health aide, and must have known the patient for at least 6 months.
Ponesimod (sponsored by Actelion Pharmaceuticals Ltd.) Protocol #: ACT-128800 Principal Investigator: Donna Graves, MD Coordinator: Maryanne Burdette – 704-446-1925	The primary objective of the study is to assess whether the drug ponesimod is superior to teriflunomide (Aubagio®) in reducing the frequency of relapses in subjects with relapsing-multiple sclerosis (RRMS). Secondary objectives are to determine if ponesimod is superior to teriflunomide in reducing inflammatory disease activity as measured by magnetic resonance imaging (MRI) technique in a subset of subjects; to assess the effect of ponesimod on other aspects of multiple sclerosis (MS) disease control, and to assess the safety and tolerability of ponesimod in subjects with relapsing-remitting multiple sclerosis (RRMS). To be eligible for the study, subjects must at least be between 18 and 55; be diagnosed with MS with or without relapse; have at least 1 relapse within the past 12 months, but not within the past 30 days. And they must have an EDSS score between 0 and 5.5, inclusive.
Tecfidera (sponsored by Biogen Idec Protocol #: 109MS308 Principal Investigator: Jill Conway, MD Coordinator: Joyce Pitner – 704-446-1349	The goal of this study is to assess the efficacy of the drug BG00012 in delaying disability progression in subjects with secondary progressive multiple sclerosis (SPMS) who are progressing independent of relapses. The drug is currently marketed for the treatment of relapsing MS. This is the first trial to measure the effectiveness of BG00012 in people with SPMS. Subjects will be randomized to receive either BG00012 or placebo (which looks like the BG00012 capsule but does not have any active ingredient). Each person will have a 50-50 chance of being placed into either group. Both groups will taking one 240-mg capsule twice daily. Individuals with SPMS may be eligible for the study if they are between 18 to 58 years old, inclusive; experienced the onset of SPMS at least 2 years before; have documented, confirmed evidence of disease progression independent of clinical relapses over the previous year; have an EDSS score of 3.0 to 6.5, inclusive; have an MS Severity Score (MSSS) of 4 or higher. Study participation will last for 108 weeks.

ARPEGGIO (sponsored by TEVA Pharmaceuticals) Protocol #: TV5600-CNS-20006 Principal Investigator: Jill Conway, MD Coordinator: Megan Kramer – 704-446-0803	Dr. Jill Conway is conducting a research study of the investigational drug laquinimod to find out whether laquinimod is safe and effective in treating people with primary progressive multiple sclerosis (PPMS). Specifically, the study will test if laquinimod reduces the loss of brain volume over time; a reduction in brain volume and reduction in the size of the brain are thought to be related to worsening of MS. Laquinimod is a new oral drug that is being developed for the treatment of MS and other indications. It is “investigational” because it has not been approved by regulatory authorities, such as the Food and Drug. This is the first study that evaluates laquinimod in patients with PPMS. Potentially eligible participants must be between ages 25 and 55, inclusive; have a confirmed diagnosis of PPMS; show disease progression in the previous 2 years and have an EDSS score between 3 and 6.5. Approximately 375 patients will take part in the study at 120 sites in the U.S., Canada and Europe, including 3-5 patients at CMC-Neurology. Participation is due to last 18-30 months.

Non-Enrolling Studies

Non-enrolling Studies

Study and Contact Information	Description
EXPEDITION (sponsored by Eli Lilly) Protocol #: H8A-MC-LZAX Principal Investigator: Oleg V. Tcheremissine, MD Coordinator: Dineen Garnder – 704-446-7511 and Megan Kramer – 704-446-0803	The purpose of this study is to determine the safety and effectiveness of the investigational drug solanezumab. Specifically, researchers want to know if solanezumab will slow mental and functional decline in individuals with mild Alzheimer’s. About 2,100 people will enroll in the study. Each person will be assigned to one of two study groups using a chance process (like flipping a coin). One group will receive solanezumab and the other, placebo. Placebo looks like the study drug but does not contain any active ingredient. Approximately 10 people are expected to be involved in this research study at CMC-Neurology, and participation will last for 18 months. Potential participants must be 55-90 years old, and meet a variety Alzheimer’s-related mental and cognitive criteria based on established medical standards.

Study and Contact Information	Description
Opexa (sponsored by Opexa Therapeutics) Protocol #: 2012-00 Principal Investigator: Jill Conway, MD Coordinator: Cynthia Lary – 704-446-6063	The purpose of this study is to evaluate the safety and effectiveness of Tcelna, an investigational T-cell (immune cell) product, compared to placebo (a substance that looks like Tcelna, but does not contain any active ingredient) in people with SPMS. An investigational product is one that is not yet approved by the U.S. Food and Drug Administration (FDA). The T-cell product being studied is manufactured for each person individually, based on each person’s specific T-cell profile. Tcelna is designed to reduce or prevent the autoimmune attack (an attack by the body’s own cells) against the nervous system (brain and spinal cord) that is believed to cause MS or make it worse over time. In this study, we want to assess whether or not Tcelna is safe and effective in reducing the signs of SPMS. The primary way we will assess whether Tcelna is effective is to measure the change in the size of the brain over time.

Study and Contact Information	Description
CD FLEX (sponsored by Merz Pharmaceuticals, LLC) Protocol #: MUS 60201 4073 1 Principal Investigator: Sanjay Iyer, MD Coordinator: Maryanne Burdette – 704-446-1925	This study compares shorter treatment intervals (Short Flex dosing) of the drug, Xeomin, a botulinum toxin treatment, to the standard treatment intervals (Long Flex dosing) of Xeomin. The goal is to determine if more frequent dosing leads to development of botulinum toxin resistance (making treatment with botulinum toxin less effective). Study participation lasts for 12-29 months. Approximately 250 subjects at 50-75 treatment centers in the United States and Canada will be enrolled.

Study and Contact Information	Description
EXTEND (sponsored by Biogen Idec) Protocol #: 205MS303 Principal Investigator: Jill Conway, MD Coordinator: Cynthia Lary – 704-446-6063	The purpose of this study is to give eligible patients who completed Study 205MS301 the opportunity to switch from Avonex to DAC HYP or to continue treatment with DAC HYP for approximately 3 more years or until the study drug is commercially available, whichever happens first. This study also allows researchers to learn more about the safety and effectiveness of long-term treatment with DAC HYP in people with multiple sclerosis (MS) and whether receiving DAC HYP treatment for a long time causes your body to develop antibodies against DAC HYP.

Study and Contact Information	Description
ENHANCE (sponsored by Biogen Idec) Protocol #: 218MS305 Principal Investigator: Donna Graves, MD Coordinator: Joyce Pitner – 704-446-1349	This research aims to examine how the drug prolonged-release fampridine affects aspects of walking and balance that have not been studied previously in patients with multiple sclerosis. There is an optional DNA research sub-study in which participants in the main study may also take part. Potentially eligible participants must be 18-70 years old and be diagnosed with primary-progressive, secondary-progressive, progressive-relapsing, or relapsing-remitting MS. Approximately 590 individuals with MS will be enrolled in the study; 6 persons will participate at CMC-Neurology, and participation will last 24 weeks.

Study and Contact Information	Description
FTY720 (sponsored by Novartis) Protocol # 2306E1 Principal Investigator: Jill Conway, MD Coordinator: Maryanne Burdette – 704-446-1925	This is the extension phase of the original FTY720-2306 study. The main purpose of this extension study is to continue to evaluate how safe and well tolerated the drug fingolimod is over the long-term in patients with PPMS who previously participated in the INFORMS core study. Other purposes are to evaluate efficacy of fingolimod over the long-term. This study is open only to patients who participated in and completed the original study.

Study and Contact Information	Description
Ascend (sponsored by Biogen Idec) Protocol #: 101MS326 Principal Investigator: Jill Conway, MD Coordinator: Maryanne Burdette – 704-446-1925	The main purpose of this research study is to see if natalizumab is effective in slowing the progression of disability independently of relapse in SPMS. Blood and urine samples will also be obtained and saved for future research on MS and on a virus called JC virus that sometimes causes infections in people taking natalizumab and other MS drugs. Finally, this study will test blood samples for biomarkers, which are naturally-occurring substances in the body, to try to learn more about how they change in MS and how they might be used to predict a patient’s response to treatment with natalizumab.

Study and Contact Information	Description
Oratorio (sponsored by F. Hoffman-LaRoche Ltd.) Protocol #: WA25046 / D Principal Investigator: Jill Conway, MD Coordinator: Cynthia Lary, 704-446-6063	The purpose of this study is to investigate the efficacy of ocrelizumab compared with placebo in patients with primary progressive multiple sclerosis, as measured by the time to onset of sustained disability progression over the treatment period, defined as an increase in EDSS that is sustained for at least 12 weeks, based on regularly scheduled visits. Also evaluating the time to sustained disability progression over the treatment period, defined as an increase in EDS S that is sustained for at least 24 weeks, the change in a timed 25-foot walk from baseline to Week 120, the change in total volume of T2 lesions on MRI scans of the brain from baseline to week 120, and to evaluate the safety and tolerability of ocrelizubab in patients with primary progressive multiple sclerosis as compared with placebo.

Study and Contact Information	Description
Mental Fatigue (sponsored by Biogen Idec) Protocol #: None Principal Investigator: Sanjay Iyer, MD Coordinator: Bridget Loven – 704-446-1987	The purpose of this study is to determine if mentally fatigued patients will show a greater and faster decline in cognition over time. Because cognitive impairment impacts so many MS patients, we need an accurate way of predicting which patients will suffer a faster decline in their ability to think, concentrate, understand instructions, reason and accomplish minor tasks so we can start therapy early. By doing this study, we also hope to develop an inexpensive, complete, and reliable way to measure cognition in MS patients.

Study and Contact Information	Description
Stratify 2 (sponsored by Biogen Idec) Protocol #: 101JC402 Principal Investigator: Sanjay Iyer, MD Coordinator: Bridget Loven – 704-446-1987	The purpose of this study is to determine whether antibodies to JC Virus (JCV) may be used to predict whether a patient is at higher or lower risk for developing Progressive Multifocal Leukoencephalopathy (PML).

Study and Contact Information	Description
Opera (sponsored by F. Hoffmann-La Roche, Ltd.) Protocol #: WA21092 Principal Investigator: Jill Conway, MD Coordinator: Joyce Pitner – 704-446-1349	The purpose of this study is to determine whether antibodies to JC Virus (JCV) may be used to predict whether a patient is at higher or lower risk for developing Progressive Multifocal Leukoencephalopathy (PML).